Preclinical Advances of Therapies for Laminopathies

Laminopathies are a group of rare disorders due to mutation in <i>LMNA</i> gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for lamin...

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Autores principales: Louise Benarroch, Enzo Cohen, Antonio Atalaia, Rabah Ben Yaou, Gisèle Bonne, Anne T Bertrand
Formato: article
Lenguaje:EN
Publicado: MDPI AG 2021
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Acceso en línea:https://doaj.org/article/1dac7776c7c14d8c90553a1911ea9631
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spelling oai:doaj.org-article:1dac7776c7c14d8c90553a1911ea96312021-11-11T17:30:03ZPreclinical Advances of Therapies for Laminopathies10.3390/jcm102148342077-0383https://doaj.org/article/1dac7776c7c14d8c90553a1911ea96312021-10-01T00:00:00Zhttps://www.mdpi.com/2077-0383/10/21/4834https://doaj.org/toc/2077-0383Laminopathies are a group of rare disorders due to mutation in <i>LMNA</i> gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for laminopathies are not fully understood, several therapeutic approaches have been evaluated in patient cells or animal models, ranging from gene therapies to cell and drug therapies. This review is focused on these therapies with a strong focus on striated muscle laminopathies and premature ageing syndromes.Louise BenarrochEnzo CohenAntonio AtalaiaRabah Ben YaouGisèle BonneAnne T BertrandMDPI AGarticle<i>LMNA</i>lamin A/CprogerinlaminopathytreatmenttherapyMedicineRENJournal of Clinical Medicine, Vol 10, Iss 4834, p 4834 (2021)
institution DOAJ
collection DOAJ
language EN
topic <i>LMNA</i>
lamin A/C
progerin
laminopathy
treatment
therapy
Medicine
R
spellingShingle <i>LMNA</i>
lamin A/C
progerin
laminopathy
treatment
therapy
Medicine
R
Louise Benarroch
Enzo Cohen
Antonio Atalaia
Rabah Ben Yaou
Gisèle Bonne
Anne T Bertrand
Preclinical Advances of Therapies for Laminopathies
description Laminopathies are a group of rare disorders due to mutation in <i>LMNA</i> gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for laminopathies are not fully understood, several therapeutic approaches have been evaluated in patient cells or animal models, ranging from gene therapies to cell and drug therapies. This review is focused on these therapies with a strong focus on striated muscle laminopathies and premature ageing syndromes.
format article
author Louise Benarroch
Enzo Cohen
Antonio Atalaia
Rabah Ben Yaou
Gisèle Bonne
Anne T Bertrand
author_facet Louise Benarroch
Enzo Cohen
Antonio Atalaia
Rabah Ben Yaou
Gisèle Bonne
Anne T Bertrand
author_sort Louise Benarroch
title Preclinical Advances of Therapies for Laminopathies
title_short Preclinical Advances of Therapies for Laminopathies
title_full Preclinical Advances of Therapies for Laminopathies
title_fullStr Preclinical Advances of Therapies for Laminopathies
title_full_unstemmed Preclinical Advances of Therapies for Laminopathies
title_sort preclinical advances of therapies for laminopathies
publisher MDPI AG
publishDate 2021
url https://doaj.org/article/1dac7776c7c14d8c90553a1911ea9631
work_keys_str_mv AT louisebenarroch preclinicaladvancesoftherapiesforlaminopathies
AT enzocohen preclinicaladvancesoftherapiesforlaminopathies
AT antonioatalaia preclinicaladvancesoftherapiesforlaminopathies
AT rabahbenyaou preclinicaladvancesoftherapiesforlaminopathies
AT giselebonne preclinicaladvancesoftherapiesforlaminopathies
AT annetbertrand preclinicaladvancesoftherapiesforlaminopathies
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